Lipidic Systems for In Vivo siRNA Delivery
Journal Title: The AAPS Journal - Year 2009, Vol 11, Issue 4
Abstract
The ability of small-interfering RNA (siRNA) to silence specific target genes not only offers a tool to study gene function but also represents a novel approach for the treatment of various human diseases. Its clinical use, however, has been severely hampered by the lack of delivery of these molecules to target cell populations in vivo due to their instability, inefficient cell entry, and poor pharmacokinetic profile. Various delivery vectors including liposomes, polymers, and nanoparticles have thus been developed in order to circumvent these problems. This review presents a comprehensive overview of the barriers and recent progress for both local and systemic delivery of therapeutic siRNA using lipidic vectors. Different strategies for formulating these siRNA-loaded lipid particles as well as the general concern about their safe use in vivo will also be discussed. Finally, current advances in the targeted delivery of siRNA and their impacts on the field of RNA interference (RNAi)-based therapy will be presented.
Authors and Affiliations
Sherry Y. Wu, Nigel A. J. McMillan
Keywords
Related Articles
- EP ID EP681505
- DOI 10.1208/s12248-009-9140-1
- Views 79
- Downloads 0
Peptide Amphiphile Micelles Self-Adjuvant Group A Streptococcal Vaccination
Delivery system design and adjuvant development are crucially important areas of research for improving vaccines. Peptide amphiphile micelles are a class of biomaterials that have the unique potential to function as both...
Hemoglobin-derived Peptides as Novel Type of Bioactive Signaling Molecules
Most bioactive peptides are generated by proteolytic cleavage of large precursor proteins followed by storage in secretory vesicles from where they are released upon cell stimulation. Examples of such bioactive peptides...
A Technique to Estimate In Vivo Dissolution Profiles Without Data from a Solution
Dissolution tests are expected to ensure adequate in vivo product performance. Given the recent progress in the ability to predict of human permeability, it is possible to synthesize the plasma drug concentration–...
Physiochemical and Biochemical Factors Influencing the Pharmacokinetics of Antibody Therapeutics
Monoclonal antibodies are increasingly being developed to treat multiple disease areas, including those related to oncology, immunology, neurology, and ophthalmology. There are multiple factors, such as charge, size, neo...
Electronic and resonance effects on the lonization of structural analogues of efavirenz
The solubility of 4 analogues of efavirenz was studied as a function of pH. The study evaluated the ionization behavior and determined the relative contribution of electronegative substituents versus resonance effects on...